UK's Evox Gets Lilly Onboard For CNS Exosome Collaboration
Banks $20m Up Front
Executive Summary
The US major will work with Evox Therapeutics on potential treatments of neurological disorders using the Oxford-based firm's DeliverEX platform.
Less than three months after linking up with Takeda Pharmaceutical Co. Ltd., the UK's Evox Therapeutics Ltd. has bagged another big pharma partner and signed a multi-target RNA interference (RNAi) and antisense pact looking at potential treatments for neurological disorders with Eli Lilly & Co..
As part of the research collaboration and five-target license agreement, Evox will be responsible for engineering exosomes using its DeliverEX platform to achieve brain/CNS-targeting, drug loading and analytics. The Oxford-headquartered biotech will also develop in vitro assays and material for initial in vivo studies, with any resulting candidates handed over to Lilly after preclinical proof-of-concept studies have been completed.
Cash-wise, Evox will receive a $20m upfront fee, research funding over three years and a $10m investment from Lilly in exchange for a convertible bond. It will also be eligible for potential milestones of up to $1.2bn, if all five unspecified neurological disease targets make it to market, plus tiered royalties up to low double digits.
CEO Tony de Fougerolles told Scrip that getting a partner of the calibre of Lilly, with its RNAi and oligonucleotide expertise, reflected well on Evox and "we're happy that they provide the drug so we don't have to worry about designing our own. We could do that but it makes more sense for them to do it." He added that evaluating the ability to apply oligonucleotide payloads to treat broad neurological diseases was “something we probably wouldn't have done ourselves just because of the cost."
De Fougerolles argued that "it makes a lot of sense for us to leverage their expertise in neurology. They bring in the cargo, we load the cargo and we do a bunch of the early preclinical characterization, then they really take it on." He said the deal represented "a very neat way to develop drugs with a world-class partner and at the same time continue to build and improve the DeliverEx platform so that we can at some point down the road use it for ourselves for something different."
DeliverEX uses the unique tissue-targeting properties of exosomes, extracellular membrane-bound vesicles that are used by cells to transport molecules around the body, Evox claims. The firm believes that exosome-based drugs "have the potential to address some of the limitations of protein, antibody and nucleic acid-based therapies by enabling delivery to cells and tissues that are currently out of reach using other drug delivery technologies."
The Lilly deal follows an agreement signed with Takeda in March potentially worth $882m which is focusing on the development of up to five novel protein replacement and mRNA therapies, including Evox's preclinical candidate for Niemann-Pick disease type C. That deal included $44m in upfront fees, near-term milestones and research funding.
The curriculum vitae of de Fougerolles is an impressive one. Prior to joining Evox, he was chief scientific officer at Ablynx NV, acquired in 2018 by Sanofi, and founding CSO ("I was employee number three") at Moderna Inc., pioneering modified messenger RNA as a new therapeutic modality. Before that, he was head of research ("employee number five") at Alnylam Pharmaceuticals Inc. where he oversaw the development of several RNAi drugs.
At Evox, "our own goal beyond doing these partnerships with Lilly and Takeda is to develop our own proprietary pipeline of drugs, bring them into clinical trials and ultimately commercialize some of them ourselves, so very similar to what I've done before at Alnylam, Moderna and Ablynx," he told Scrip. The company's own lead candidate is an exosome-based therapy to treat argininosuccinic aciduria (ASA), a rare metabolic disease, and de Fougerolles said that an investigational new drug (IND) filing to begin clinical trials was likely in the next 18-24 months.
He said that behind the ASA program, "we've got several other rare disease programs of our own that we're advancing which we haven't disclosed yet."
As to whether more deals along the lines of the Lilly and Takeda pacts are in the offing, de Fougerolles answered, "There are some strategic fields where we still see working with a partner would make a lot of sense and we're having early discussions around some of those other places where a partner brings its expertise, we bring ours and the sum adds up to more than the parts. That being said, we also want to be selective around that because we want to make sure that we really invest a lot in our own pipeline, in the long term that's where the real validation of the technology lies."
The exosome-based therapy area is in its infancy but de Fougerolles noted that Evox, founded in 2016, "was one of the first ones in the space and we've got really comprehensive and pretty dominating intellectual property" covering the key applications of exosome therapeutics, including delivery of RNA and protein-based drugs and manufacturing.